EuropaBio Information Day

1
EuropaBio Information Day

• The Invisible Revolution
• Looking back, looking forward

2
Michael Griffith

• Chief Executive
• Fighting Blindness
• Chairman
• European Platform for Patients Organisations,
  Science and Industry

3
Learning from Rare Diseases the Potential for
Change

• May 30th 2006

4
Michael Griffith

• Chief Executive, Fighting Blindness
• Chairman, EPPOSI
• Chairman, Debra Ireland
• Chairman, IPPOSI
• Chairman, MRCG
• Chairman, C4C
• Management Committee, Retina Intl.
• Executive Committee, Debra International

5

6
What can we learn from Rare Diseases

• 46 of all Bio Pharmaceuticals authorised by FDA
  between 1995 and 2000 were for rare diseases
• Reduced a little but over the past 5 years 30
  of newly authorised Biologics have been Orphan
  Products
• Marlene Haffner, Director of Office Orphan
  Products Development, FDA

7

• Similar Position in Europe

8
Status of Orphan Applications April 2006
9
Status of Orphan Applications
10
Biotech Products in Orphan Applications
11
Future

• Expect this trend to continue and increase
  common diseases breaking down into sub groups
  (personalized medicines)
• Many drugs authorized for rare diseases have
  become blockbusters e.g.Epogen/Amgen and
  Procrit/Ortho Biotech - 5b pa 2001)
• Many people now express doubt about big Pharma
  blockbuster search approach

12
Whats Different about Rare Diseases?

• Logical approach to therapy development
• Basic research to establish cause of disease
• Development of method to address that cause
  (intelligent drug design)

13
Economics

• Latest cost of drug development 2 billion
• Escalating at incredible speed
• Unsustainable in longer term

14
Economics (2)

• Now feel intelligent drug design approach will
  become the norm, even for big Pharma
• Economics are very challenging
• What can we learn from rare diseases?

15
Large Multinational Biotech Companies

• Many Biotech companies which started in Rare
  Diseases have become huge multinationals
• e.g.
• Amgen
• Genentech
• Genzyme

16
Learning from Rare Diseases

• Orphan Medicinal Products Regulation / Orphan
  Drugs Act
• Committee for Orphan Medicinal Products
• Several examples of Patient Groups working
  closely with Industry e.g. Pompe Disease
  associations working with Pharming/Genzyme
  (Myosyme)

17
Learning from Rare Diseases (2)

• Patients supported basic research, clinical
  research discussions with regulators and now
  reimbursers.
• No reason why this type of approach cannot exist
  for common diseases
• Developments in this regard already e.g. Proposed
  Patient Committees at EMEA for Paediatric
  Medicines Advanced Medicines

18
Patient Revolution

• Rare Disease therapy development has taught us
  that the way forwards is to work with Patient
  Groups
• Patient revolution is happening (recent book by
  Simon Roosendaal)

19
Patient Revolution (2)

• Appointment of 2 Patient Representatives to main
  Board of EMEA
• Patient representation must be by patient groups,
  not by research groups studying patients or by
  insurers or sick funds

20
Patient Revolution (3)

• Patients and industry must work together to
  ensure equitable and timely access to beneficial
  innovation
• Involvement of patients in consensus building in
  difficult societal issues such as the value of
  innovation, reimbursement, health care budgeting,
  access etc, is essential

21
Funding Patient Groups

• All this must be recognised and underpinned by
  funding from EU as patient groups are working on
  shoestring budgets
• Such funding as they get often comes from
  industry

22
Winston Churchill

• Some regard private enterprise as if it were a
  predatory tiger to be shot. Others look upon it a
  s a cow that can be milked. Only a handful see it
  for what it really is the strong horse that
  pulls the whole cart

23
EU Funding

• The EU Commission seems to oscillate between the
  tiger and the cow.
• It should recognise that at best industry can
  only part fund patient organisations
• Face its responsibility to fund these groups.

24
Conclusion

• We can learn better ways to develop treatments
  from seeing what has happened over the last 25
  years in Rare Diseases.
• Essential to work with patient groups
• Essential for EU to fund these groups.